The Food and Drug Administration (FDA) has approved the Investigational New Drug (IND) application for the phase 2a study of opaganib (RedHill Biopharma) in patients with confirmed moderate to severe SARS-CoV-2 infection.

Opaganib is an investigational, first-in-class, oral, sphingosine kinase-2 (SK2) selective inhibitor with anticancer, anti-inflammatory and antiviral properties. Preclinical data have shown the potential of opaganib to inhibit viral replication and reduce levels of pro-inflammatory cytokines interleukin-6 and tumor necrosis factor-alpha, which have been found to be elevated in patients with coronavirus disease 2019 (COVID-19).

The double-blind, placebo-controlled phase 2a study will enroll up to 40 patients with moderate to severe COVID-19 and pneumonia who require hospitalization and supplemental oxygenation. Patients will be randomized to receive either opaganib or placebo in addition to standard of care. The primary end point is the reduction in total oxygen requirements over the course of the 14 day treatment period. Secondary end points include time to 50% reduction in oxygen requirements, as well as the proportion of patients without fever and with negative nasal swabs, both at day 14.

“We are pleased to offer opaganib to hospitalized patients as part of a clinical study and are hopeful to meet the strong unmet need for treatments to decrease the severity and duration of respiratory symptoms due to COVID-19,” said Kevin Winthrop, MD, MPH, Professor of Infectious Diseases and Public Health at the OHSU-PSU School of Public Health and Principal Investigator of the study.

Preliminary findings from 6 hospitalized patients showed that treatment with opaganib under compassionate use resulted in decreased supplemental oxygenation requirements, higher lymphocyte counts and decreased C-reactive protein (CRP) levels within days of treatment initiation. Clinical improvement was observed in patients with and without hydroxychloroquine. Regarding safety, opaganib was found to be well tolerated.

All 6 patients have since been weaned from oxygen and have been discharged from the hospital, according to the Company.